2024
Next generation triplex-forming PNAs for site-specific genome editing of the F508del CFTR mutation
Gupta A, Barone C, Quijano E, Piotrowski-Daspit A, Perera J, Riccardi A, Jamali H, Turchick A, Zao W, Saltzman W, Glazer P, Egan M. Next generation triplex-forming PNAs for site-specific genome editing of the F508del CFTR mutation. Journal Of Cystic Fibrosis 2024 PMID: 39107154, DOI: 10.1016/j.jcf.2024.07.009.Peer-Reviewed Original ResearchCystic fibrosis transmembrane conductance regulatorCystic fibrosis transmembrane conductance regulator geneF508del-CFTR mutationPeptide nucleic acidCFBE cellsBronchial epithelial cellsCystic fibrosisTriplex-forming peptide nucleic acidsDonor DNACFTR mutationsEpithelial cellsCFTR functionMutations associated with genetic diseasesPrimary nasal epithelial cellsAnalysis of genomic DNAGenetic diseasesIncreased CFTR functionDevelopment of peptide nucleic acidsImprove CFTR functionTransmembrane conductance regulatorAutosomal recessive genetic diseaseNasal epithelial cellsAir-liquid interfaceCystic fibrosis bronchial epithelial cellsHuman bronchial epithelial cells
2022
Surface conjugation of antibodies improves nanoparticle uptake in bronchial epithelial cells
Luks VL, Mandl H, DiRito J, Barone C, Freedman-Weiss MR, Ricciardi AS, Tietjen GG, Egan ME, Saltzman WM, Stitelman DH. Surface conjugation of antibodies improves nanoparticle uptake in bronchial epithelial cells. PLOS ONE 2022, 17: e0266218. PMID: 35385514, PMCID: PMC8986008, DOI: 10.1371/journal.pone.0266218.Peer-Reviewed Original ResearchConceptsTarget-specific antibodiesNanoparticle uptakeSurface conjugationNanoparticle surface modificationSurface of nanoparticlesCellular uptakeSite-specific geneSpecific cellular bindingNanoparticlesIntracellular deliveryEditing reagentsBronchial epithelial cellsSurface modificationCellular targetingCystic fibrosisTherapeutic agentsEpithelial cellsParticle uptakeFeasible strategyGenetic diseasesFirst demonstrationHuman bronchial epithelial cellsKinetics of antibodiesCellular bindingAppropriate antibodies
1992
Defective regulation of outwardly rectifying Cl− channels by protein kinase A corrected by insertion of CFTR
Egan M, Flotte T, Afione S, Solow R, Zeitlin P, Carter B, Guggino W. Defective regulation of outwardly rectifying Cl− channels by protein kinase A corrected by insertion of CFTR. Nature 1992, 358: 581-584. PMID: 1380129, DOI: 10.1038/358581a0.Peer-Reviewed Original ResearchConceptsCyclic AMP-dependent proteinCF geneCF bronchial epithelial cellsLethal genetic diseaseProtein kinaseDefective acidificationBronchial epithelial cellsGenetic diseasesProteinDefective regulationLarge conductanceEpithelial cellsGenesCFTRConductance pathwayCystic fibrosisLinear current-voltage relationshipVirus vectorsRegulationKinaseConductancePathwayExpressionAcidificationCells